Sankt Augustin

Researchers are evaluating the long-term safety of subcutaneous guselkumab in children with moderately to severely active ulcerative colitis, Crohn's disease, or juvenile psoriatic arthritis. This Phase 3, open-label study aims to monitor the safety of this treatment over an extended period in a pediatric population. Participants will receive guselkumab through subcutaneous injections. The study includes those who have completed the initial pediatric guselkumab dosing and have benefited from continued therapy as judged by their doctor. The study focuses on long-term treatment, with safety assessed by tracking adverse events for up to 6 years and 9 months. During the study, children will be regularly monitored for treatment-emergent adverse events. Parents or guardians will provide consent, and children able to understand will give assent. Researchers will collect data to assess safety throughout the treatment period, ensuring careful observation of participants' health and responses to guselkumab.

Researchers are conducting a phase II trial to evaluate the clinical effectiveness, safety, and tolerability of MAS825 in children and adults diagnosed with Still's disease. The study focuses on participants with active disease signs such as elevated CRP or ferritin levels, fever, rash, arthritis, serositis, or macrophage activation syndrome, who also require glucocorticoid treatment. This trial aims to gather important information about MAS825's role in managing Still's disease across a wide age range. Participants will receive MAS825, an experimental drug, in an open-label format, meaning all participants will be given the study drug without a placebo comparison. The study does not specify dosing details but monitors participants closely for response and safety. There is no mention of separate treatment arms or extension periods in the provided information. Throughout the study, researchers will assess participants' clinical response by monitoring disease activity markers and symptoms up to Day 85. Safety and tolerability will also be evaluated through regular assessments. The trial includes pediatric and adult participants aged 1 to 100 years and tracks the number of participants showing a clinical response based on predefined criteria. Participants will be monitored for adverse events, and their need for glucocorticoids will be considered during the study.

The total study duration per patient will be 166 weeks that will consist of a 4- week screening, a 12-week core treatment phase, a 144-week extension phase, and a 6-week post-treatment follow-up.

Researchers are studying whether baricitinib can help preserve beta-cell function in children and adults newly diagnosed with type 1 diabetes. This Phase 3 trial focuses on participants aged 1 to less than 36 years who have recently been diagnosed with this condition. The goal is to understand if baricitinib, compared to a placebo, can maintain insulin-producing cell activity. Participants will be randomly assigned to receive either baricitinib or a placebo, both given orally. The study is double-blind, meaning neither participants nor researchers know who receives the active drug or placebo. Treatment and observation will continue for about 60 weeks. During the study, participants will undergo evaluations including measuring C-peptide levels to assess beta-cell function at the start and after 52 weeks. Researchers will monitor health status, collect laboratory tests, and track any side effects or changes in diabetes-related markers to determine the effects of baricitinib over the study period.

Researchers are evaluating the long-term safety and effectiveness of baricitinib for treating Juvenile Idiopathic Arthritis (JIA) in children and teenagers aged 1 to less than 18 years. This phase 3 study focuses on participants who have previously taken part in baricitinib studies I4V-MC-JAHV or I4V-MC-JAHU. The goal is to monitor how well baricitinib works and how safe it is over an extended period in this young population. Participants will receive baricitinib orally as part of the study treatment. Since all participants have prior exposure to baricitinib in earlier studies, this trial continues to observe their response and any long-term effects. The study does not mention a separate comparator group or additional interventions beyond baricitinib administration. During the study, researchers will track serious adverse events and any permanent discontinuations of baricitinib from the start through week 264. Participants will be regularly monitored for safety and treatment effects throughout this long-term follow-up. This extended observation helps assess the ongoing impact of baricitinib on juvenile arthritis over several years.

Juvenile psoriatic arthritis (jPsA) is a type of arthritis that starts before age 16 and occurs when the immune system attacks healthy cells causing joint pain, stiffness, and swelling. This research aims to evaluate the safety of risankizumab for treating jPsA and to observe changes in disease symptoms. The study compares risankizumab with adalimumab, an approved treatment for jPsA, in a phase 3 clinical trial involving approximately 40 children worldwide. Participants are randomly assigned to receive either risankizumab or adalimumab as subcutaneous injections based on their body weight. The first period lasts 24 weeks, during which the assigned treatment is given. Those who respond to their treatment continue the same medication for an additional 100 weeks in the second period. If symptoms worsen during this time, participants may be withdrawn. After treatment ends, safety follow-up lasts 70 days for adalimumab and 140 days for risankizumab recipients. Throughout the study, participants attend regular visits at clinics or hospitals where medical assessments, blood tests, and questionnaires are used to monitor treatment effects and side effects. Researchers will measure improvement in arthritis symptoms and record any adverse events up to 144 weeks. The total study involvement can last over two years, including treatment and follow-up periods.

Researchers are studying the pharmacokinetics, pharmacodynamics, efficacy, and safety of anifrolumab given by intravenous infusion compared with placebo in children aged 5 to under 18 years with moderate to severe active systemic lupus erythematosus (SLE) who are also receiving standard care treatments. This Phase III trial aims to better understand how anifrolumab works and its effects in this pediatric population. The study lasts about 116 weeks and includes several parts: a screening period of up to 30 days; Part A, a four-week double-blind, placebo-controlled, randomized phase focusing on pharmacokinetics; Part B, a double-blind, placebo-controlled, randomized safety and efficacy phase lasting 48 to 52 weeks depending on prior participation; Part C, a 52-week open-label extension where all participants receive anifrolumab; and Part D, a safety follow-up visit 12 weeks after the last dose. Participants will have regular evaluations including blood tests to measure drug levels and response, assessments of lupus activity, and monitoring for side effects. Key outcomes include drug concentration levels at various times and the number of participants responding to treatment at week 52. The study carefully tracks safety and effectiveness throughout all phases, with total participation lasting nearly two and a half years.

Researchers are evaluating the plasma concentrations of bimekizumab in children aged 2 to less than 18 years who have active juvenile idiopathic arthritis, specifically enthesitis-related arthritis (including juvenile-onset ankylosing spondylitis) and juvenile psoriatic arthritis. The study aims to understand how the drug behaves in the body when given by subcutaneous injection and to assess its safety in these pediatric participants. This is an open-label, single-arm Phase 3 trial focusing on these specific arthritis subtypes. Participants will receive bimekizumab injections at predetermined times during the study. The treatment period includes an initial phase up to 16 weeks where the drug levels in plasma will be monitored. Participants already taking methotrexate or sulfasalazine may continue these stable medications, but no other disease-modifying or immunosuppressive drugs are allowed during the study. The study does not require prior use of methotrexate or sulfasalazine and excludes participants who have received certain other biologic therapies. Throughout the study, children will be assessed for active joint involvement and enthesitis, and regular plasma sampling will be done to measure bimekizumab concentrations. Safety will be closely monitored, including evaluations for infections, laboratory tests, and mental health status. The study requires informed consent/assent and follows participants for the initial treatment period and beyond to ensure thorough monitoring of drug effects and safety.

Researchers are evaluating the effectiveness of apremilast compared with a placebo in treating juvenile psoriatic arthritis (JPsA) in children aged 5 to less than 18 years. This phase 3, multicenter, double-blind, randomized, placebo-controlled study aims to assess the safety, efficacy, and drug behavior in pediatric participants with active JPsA, a condition involving arthritis and psoriasis or related symptoms. Participants will be randomly assigned to receive either oral apremilast or a matching oral placebo. The study will monitor the response to treatment over a 16-week period, focusing on achieving a specific improvement based on American College of Rheumatology Pediatric (ACR Pedi 30) criteria. Both treatments will be administered during this time to compare their effects. During the study, children will be regularly assessed for joint activity and disease symptoms. Researchers will measure the number of participants achieving the ACR Pedi 30 response by week 16 to evaluate treatment effectiveness. Safety and pharmacokinetics will also be monitored throughout, with the total participation duration covering the initial 16 weeks of treatment observation.

Researchers are conducting a multinational clinical trial called LBL 2018 to study treatment approaches for children and adolescents newly diagnosed with lymphoblastic lymphoma. The trial aims to see if replacing prednisone with dexamethasone in induction therapy can reduce relapse involving the central nervous system (CNS) and if intensified treatment improves event-free survival in high-risk patients. Participants are grouped into high risk and standard risk categories based on disease characteristics to guide treatment and randomization. The study compares standard chemotherapy treatment using prednisone to an experimental approach using dexamethasone during induction. Patients in standard risk groups receive induction, consolidation, extra-compartment phases, reintensification (for some), and maintenance therapy over 24 months. High-risk patients can be randomized again after induction to receive either standard or intensified chemotherapy including additional PEG asparaginase and alternating high-risk courses. Patients with CNS involvement receive intensified intrathecal therapy and additional doses during maintenance without cranial irradiation. Participants undergo treatment phases lasting up to 24 months with follow-up for up to 7.25 years to monitor relapse rates and event-free survival. Evaluations include disease status, CNS involvement, and response to therapy. The trial collects pathology and genetic samples for risk stratification and monitors safety and treatment effects across multiple international centers. This comprehensive study seeks to improve outcomes while reducing CNS relapses in young patients with lymphoblastic lymphoma.